A Phase 3, Randomized, Open-Label, Multicenter Study to Evaluate the Safety (Compared to Iron Sucrose), Efficacy and Pharmacokinetics of Ferumoxytol for the Treatment of Iron Deficiency Anemia (IDA) in Pediatric Subjects With Chronic Kidney Disease (CKD)
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 129
- 试验地点
- 29
- 主要终点
- Hemoglobin change of at least 0.5 g/dL from Baseline to Week 5
研究概览
简要总结
Primary Objectives:
To evaluate the safety (compared to iron sucrose) and efficacy of ferumoxytol in pediatric CKD subjects with iron deficiency anemia (IDA) or who are at risk of development of IDA
Secondary Objective:
To determine the single-dose pharmacokinetics (PK) and pharmacodynamics (PD) profile of ferumoxytol in pediatric subjects.
详细描述
This is a Phase 3, randomized, open-label, multicenter, study of the safety (compared to iron sucrose), efficacy, and PK/PD of ferumoxytol (7.0 mg Fe/kg x 2 [max 510 mg/dose]) in pediatric subjects with iron deficiency anemia (IDA) and CKD. There will be a total of approximately 125 subjects randomized to treatment in a 2:1 ratio to either ferumoxytol or iron sucrose.
Total subject participation in the study will be up to 7 weeks, which includes a 2-week Screening Period and a 5-week Treatment Period.
Subjects receive the following:
• Two IV infusions of ferumoxytol 7.0 mg Fe/kg (max 510 mg/dose), the first administered on Day 1 and the second 2-8 days later
OR
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female 2 years to <18 years of age at time of consent
- •Has IDA defined as: a) hemoglobin <12.0 g/dL and b) with either transferrin saturation (TSAT) <40% or ferritin <100 ng/mL; or considered to be at risk of development of IDA, i.e., TSAT<20% with falling hemoglobin during the preceding 2 months and a history of hemoglobin <12 g/dL
- •Has Chronic Kidney Disease defined as one of the following:
- •on chronic hemodialysis;
- •receiving chronic peritoneal dialysis;
- •estimated glomerular filtration rate (eGFR) of <60 mL/min/1.73 m2;
- •has evidence of structural and/or functional abnormalities e.g., persistent albuminuria, abnormal urine sediment, electrolyte and other abnormalities due to tubular disorders for > 3 months.
- •For patients other than hemodialysis dependent CKD patients, documented history of unsatisfactory oral iron therapy or in whom oral iron cannot be tolerated, or for whom oral iron is considered medically inappropriate
- •All subjects (female and male) of childbearing potential who are sexually active must be on an effective method of birth control for at least 1 month prior to Day 1 Dosing and agree to remain on birth control until completion of the study
排除标准
- •Known hypersensitivity reaction to any component of ferumoxytol and iron sucrose
- •History of allergy to intravenous (IV) iron
- •History of multiple drug allergies (>2)
- •Low systolic blood pressure (Age 1-9 years <70 + [age in years x 2] mmHg, Age 10-17 years <90 mmHg)
- •Hemoglobin ≤7.0 g/dL
- •Serum ferritin level >600 ng/mL
研究组 & 干预措施
Ferumoxytol
干预措施: Ferumoxytol (Drug)
Iron sucrose
干预措施: Iron sucrose (Drug)
结局指标
主要结局
Hemoglobin change of at least 0.5 g/dL from Baseline to Week 5
时间窗: 35 days
Proportion of patients achieving a hemoglobin change of at least 0.5 g/dL during the period from Baseline to Week 5
次要结局
- Incidence of adverse events of special interest (AESI)(49 days)
- Incidence of Treatment Emergent Adverse Events(49 days)